Open-label Safety and Efficacy Evaluation of Fx-1006a in Patients with V122i Or Wild-type Transthyretin (ttr) Amyloid Cardiomyopathy. Patients who successfully complete Fx1B-201 will report to the clinical unit on Day 0 to sign the informed consent form and determine eligibility for Protocol Fx1B-303. In addition, on Day 0, patients will have their entrance criteria reviewed, and medical histories and demographic characteristics obtained. The physical examination (including weight and vital signs) and the relevant end of study clinical laboratory tests (alkaline phosphatase, alanine aminotransferase, aspartate aminotransferase, blood urea nitrogen, gamma glutamyl transferase, creatinine, total bilirubin, international normalized ratio, troponin I, troponin T, and amino-terminal B-type natriuretic peptide) from Protocol Fx1B-201 will be used for Protocol Fx1B-303. If more than 30 days has elapsed between the final study visit of Protocol Fx1B-201 and Day 0 of Protocol Fx1B-303, an abbreviated physical examination (including weight and vital signs) and clinical laboratory assessments must be performed on Day 0. Eligible patients will begin once-daily dosing with 20 mg Fx-1006A at home on Day 1 (i.e., first dose) and will return to the clinical unit for study visits every 6 months. Adverse events (AEs) and concomitant medication use will be collected at each 6-month visit to the clinical unit. Blood draws for clinical safety laboratory tests and abbreviated physical examinations (including weight and vital signs) will also be performed at each 6-month clinic visit. ECGs will be performed every 12 months on an annual basis. A telephone call will be made at 3-month intervals between clinic visits to assess safety and use of concomitant medications. For the evaluation of efficacy, the Patient Global Assessment, NYHA classification, KCCQ, 6-minute walk test, and efficacy-related clinical laboratory tests (serum levels of troponin T, troponin I, and NT-pro-BNP) will be determined every 6 months. In addition, echocardiograms will be performed every 12 months on an annual basis. An end of study visit including all safety and efficacy assessments will occur upon patient completion of the study, premature withdrawal (for any reason), or in the event of program discontinuation by the Sponsor.
Age range
18 Years – 75 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
Percentage of Participants With Categories of Patient Global Assessment (PGA) for Follow-up Visit: Month 12
Timeframe: Month 12
Percentage of Participants With Categories of Patient Global Assessment (PGA) for Follow-up Visit: Month 60
Timeframe: Month 60
Number of Participants Classified on the Basis of New York Heart Association (NYHA) Classification at Baseline
Timeframe: Baseline
Number of Participants With Change From Baseline in New York Heart Association (NYHA) Classification at Month 12
Timeframe: Baseline, Month 12
Number of Participants With Change From Baseline in New York Heart Association (NYHA) Classification at Month 60
Timeframe: Baseline, Month 60
Change From Baseline in Total Distance Walked During 6 Minute Walk Test (6MWT) at Month 12
Timeframe: Baseline, Month 12
Change From Baseline in Total Distance Walked During 6 Minute Walk Test (6MWT) at Month 60
Timeframe: Baseline, Month 60
Number of Participants Categorized Based on Total Distance Walked During 6 Minute Walk Test (6MWT) at Month 12
Timeframe: Month 12
Number of Participants Categorized Based on Total Distance Walked During 6 Minute Walk Test (6MWT) at Month 60
Timeframe: Month 60
Change From Baseline in Dyspnea and Fatigue Scores During 6 Minute Walk Test (6MWT) at Month 12
Timeframe: Baseline, Month 12
Change From Baseline in Dyspnea and Fatigue Scores During 6 Minute Walk Test (6MWT) at Month 60
Timeframe: Baseline, Month 60
Number of Participants Categorized on Basis of Change From Baseline in Total Distance Walked During 6 Minute Walk Test (6MWT) at Month 12
Timeframe: Baseline, Month 12
Number of Participants Categorized on Basis of Change From Baseline in Total Distance Walked During 6 Minute Walk Test (6MWT) at Month 60
Timeframe: Baseline, Month 60
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Summary Score at Month 12
Timeframe: Baseline, Month 12
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Summary Score at Month 60
Timeframe: Baseline, Month 60
Change From Baseline in Troponin I and Troponin T Serum Levels at Month 12
Timeframe: Baseline, Month 12
Change From Baseline in Troponin I and Troponin T Serum Levels at Month 60
Timeframe: Baseline, Month 60
Change From Baseline in Amino-Terminal B-type Natriuretic Peptide (NT-proBNP) Serum Level at Month 12
Timeframe: Baseline, Month 12
Change From Baseline in Serum Levels of Amino-Terminal B-type Natriuretic Peptide (NT-proBNP) at Month 60
Timeframe: Baseline, Month 60